Longo, Nicola http://orcid.org/0000-0002-3677-1216
Dimmock, David
Levy, Harvey
Viau, Krista
Bausell, Heather
Bilder, Deborah A.
Burton, Barbara
Gross, Christel
Northrup, Hope
Rohr, Fran
Sacharow, Stephanie
Sanchez-Valle, Amarilis
Stuy, Mary
Thomas, Janet
Vockley, Jerry
Zori, Roberto
Harding, Cary O.
Article History
Received: 14 August 2018
Accepted: 29 November 2018
First Online: 14 December 2018
DISCLOSURE
: <b>General statement:</b> All authors of the manuscript were chosen as Steering Committee members based on their unique experience of treating patients with pegvaliase through the clinical trial program. All Steering Committee members were offered a discretional honorarium by BioMarin for their participation in the meetings and processes used to develop this guidance. An independent medical communications agency (Lucid) was employed to perform the role of methodology expert and secretariat throughout the process and to ensure editorial independence from the sponsor. Participants did not receive payment for completing the Delphi survey. Authors did not receive payment for development of the manuscript.N.L. has participated as an investigator in clinical trials sponsored by Aeglea, BioMarin Pharmaceutical Inc., Genzyme, Horizon Pharma, Protalix, Retrophin, Shire, Stealth Therapeutics, and Ultragenyx. He is on the Steering Committee for the pegvaliase clinical trial program. He has received grants from Lumos Pharma and consultancy fees from Aeglea, BioMarin Pharmaceutical Inc., Censa Pharmaceuticals, Dimension Therapeutics, Genzyme, Hemoshear, Horizon Pharma, Lumos Pharma, Moderna, Mitobridge, Pfizer, Retrophin, and Stealth Therapeutics. He has received travel fees from BioMarin Pharmaceutical Inc., Cello Health Sciences, Lumos Pharma, and SigmaTau/AlphaSigma. D.D. has (in the past 5 years) participated as an investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc., Genzyme/Sanofi, and Amicus Therapeutics. He has received research support and consultancy fees from BioMarin Pharmaceutical Inc. and Demeter Therapeutics. He is a member of the Scientific and Clinical Advisory Board at Audentes Therapeutics Inc. and has served as a consultant for Ichorion Therapeutics and Complete Genomics. H.L. is involved in clinical trials sponsored by BioMarin Pharmaceutical Inc., has acted as a consultant, and has participated in advisory boards for BioMarin Pharmaceutical Inc. K.V. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc., Horizon Pharma, and Ultragenyx. She has received consultancy fees from BioMarin Pharmaceutical Inc. and Horizon Pharma and has participated in advisory boards held by BioMarin Pharmaceutical Inc. H.B. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. and Ultragenyx. She has received consultancy fees from BioMarin Pharmaceutical Inc., Horizon Pharma, Cambrooke Therapeutics, and Nutricia. D.A.B. has been involved in a phase 3 study sponsored by BioMarin Pharmaceutical Inc., has participated in advisory boards held by BioMarin Pharmaceutical Inc. and Audentes Therapeutics Inc., and has received consultancy fees from BioMarin Pharmaceutical Inc. B.B. has participated as an investigator for clinical trials sponsored by by Alexion, BioMarin Pharmaceutical Inc., Genzyme Sanofi, Shire, and Ultragenyx Pharmaceuticals Inc. She has participated in advisory boards for BioMarin Pharmaceutical Inc., Horizon Pharma, Nestle, and Viking Therapeutics, and has received research grants from Shire and consultancy fees from Alexion, Shire, BioMarin Pharmaceutical Inc., and RegenxBio. C.G. is involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. H.N. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. and The University of Texas Health Science Center, Houston, and has participated in advisory boards held by and received consultancy fees from BioMarin Pharmaceutical Inc. F.R. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. and has received consultancy fees from BioMarin Pharmaceutical Inc. S.S. is an investor in clinical trials sponsored by BioMarin Pharmaceutical Inc. and has participated in advisory boards held by BioMarin Pharmaceutical Inc. A.S.‐V. has participated as an investigator for clinical trials sponsored by Alexion, BioMarin Pharmaceutical Inc., Genzyme, Horizon Pharma, and Ultragenyx Pharmaceuticals Inc. She has participated in advisory boards for BioMarin Pharmaceutical Inc. and Horizon Pharma. M.S. has participated as a principal investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc. J.T. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc., and has participated in advisory boards held by BioMarin Pharmaceutical Inc. She is a member of the Steering Committee for the pegvaliase clinical program. J.V. has participated as an investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc. and has received research support from BioMarin Pharmaceutical Inc. He was a speaker at the BC Childrens’ and Womens’ Hospital in Vancouver on behalf of the Canadian PKU Alliance; this event was sponsored by BioMarin Pharmaceutical Inc., Cambrooke Therapeutics, Mead Johnson, Nutricia, and Vitaflo. R.Z. has participated as an investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc. and Hyperion Therapeutics Inc. and is on the Steering Committee for the pegvaliase clinical program. C.O.H. has participated as an investigator for clinical trials sponsored by BioMarin Pharmaceutical Inc. and has received consultancy fees services and research grants from BioMarin Pharmaceutical Inc., Cydan Development Inc., Dimension Therapeutics, Horizon Pharma, Pfizer, Rubius Therapeutics, StrideBio, and Synlogic.